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Clinical Trials in the UK / NCT04159103
Recruiting Phase 1/2

Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia

NCT04159103 · tracked via the Priya Life Science UK tracker
Sponsor
ModernaTX, Inc.
Phase
Phase 1/2
Started
2021-04-15
Last updated
2026-01-22

Condition(s) studied

Propionic Acidemia

Investigational drug(s) / intervention(s)

mRNA-3927

mRNA-3927: mRNA-3927 dispersion for IV infusion

Study summary

This 3-part, Phase 1/2 study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the selected dose of mRNA-3927 in participants with genetically confirmed propionic acidemia (PA). After establishing a dose with an acceptable safety and pharmacodynamic (PD) response for participants ≥1 year of age in Part 1, participants will be enrolled in Part 2 (which will serve as the pivotal study) to allow for determination of the efficacy, safety, and PD of mRNA-3927. Part 3 will evaluate the safety, efficacy and PD response of mRNA-3927 in infants (\<1 year of age).

Eligibility

Sex
ALL
Min age
Max age
Healthy volunteers
No
Inclusion Criteria: Participants ≥1 year of age are eligible to be included in the study only if all of the following criteria apply: * ≥ 8 years of age at the time of consent/assent if enrolled as 1 of the first 2 participants in Part 1. * ≥1 year of age at the time of consent/assent if enrolled after the first 2 participants in Part 1. * Confirmed diagnosis of PA based on diagnosis by molecular genetic testing via central laboratory (PCCA and/or PCCB mutations). * Part 2 only: At least one documented MDE in the 12-month period before consent. Participants \<1 Year of Age : * Identification by newborn screening shortly after birth or having suspected PA by presenting with a spectrum of metabolic symptoms, and having a sibling diagnosed with PA. Participant may enter the Screening Period while awaiting genetic testing results, provided that all other eligibility criteria are met but would not be enrolled until diagnosis of PA is confirmed. * For infants in the neonatal intensive care unit (NICU) only: ≥37 weeks gestational age at the time of birth without other conditions/comorbidities that in the opinion of the Investigator may interfere with the interpretation of study results. * Body weight ≥3 kilograms (kg) at Screening. * At least 1 documented PA-related event prior to Screening defined as the following criteria: * Clinical signs of metabolic deterioration consistent with PA (for example, vomiting, not feeding well/poor suck, heavy breathing, lethargy, absence of proper perfusion, abnormal movements including bicycling, abnormal tone, low body temperature, seizure\[s\]), OR * Meeting the criteria of MDE definition, OR * Evidence of laboratory abnormalities as evidenced by at least one of the following: * Metabolic acidosis with elevated anion gap. * Acute hyperammonemia. * Neutropenia or thrombocytopenia. Exclusion Criteria: Participants of all ages are excluded from the study if during Screening any of the following criteria apply: * Any individual with laboratory abnormalities considered to be clinically significant (for example, markedly out of range, associated with clinical symptoms) in the Investigator or Sponsor's opinion that could interfere with or limit the participation in the study. * Estimated glomerular filtration rate (eGFR) \<30 milliliters (mL)/minute/1.73 square meter (m\^2) for participants of all ages receiving chronic dialysis. * History of organ transplantation or planned organ transplantation during the period of study participation. * Corrected QT interval (QTc) \>480 milliseconds (ms) using Bazett's correction. * Grade 3 or 4 heart failure according to the Modified Ross Heart Failure Classification for Children or the New York Heart Association Classification. * Pregnant or breastfeeding. * Other clinically significant conditions that in the Investigator's opinion could interfere with the safety of the participant, the interpretation of study results, or limit the participation in the study.

Primary outcome measure(s)

Trial sites (35)

FacilityCityRegionStatus
UCSD Altman Clinical and Transalational Research Institute Building Los Angeles California Not Yet Recruiting
Ronald Reagan UCLA Medical Center Los Angeles California Recruiting
Lucile Packard Children's Hospital Stanford Stanford California Recruiting
Nicklaus Children's Hospital Miami Florida Not Yet Recruiting
University of South Florida - 12901 Bruce B Downs Tampa Florida Not Yet Recruiting
Ann and Robert H Lurie Childrens Hospital of Chicago Chicago Illinois Recruiting
Johns Hopkins Hospital, Adult Outpatient Clinical Research Unit Baltimore Maryland Completed
Boston Children's Hospital Boston Massachusetts Completed
University of Michigan Hospitals Ann Arbor Michigan Recruiting
Icahn School of Medicine at Mount Sinai - Clinical Research Unit New York New York Recruiting
Duke University Medical System (Duke Health) Durham North Carolina Recruiting
Cincinnati Children's Hospital Medical Center Cincinnati Ohio Completed
University Hospitals Cleveland Medical Center - 11100 Euclid Ave Cleveland Ohio Active Not Recruiting
Children's Hospital of Philadelphia (CHOP) Philadelphia Pennsylvania Recruiting
Texas Children's Hospital Houston Texas Recruiting
Stollery Children's Hospital University of Alberta Edmonton Alberta Recruiting
Hospital For Sick Children Toronto Ontario Recruiting
CHU de Marseille - Hôpital de la Timone Marseille France Active Not Recruiting
Hôpital Necker - Enfants Malades Paris France Recruiting
Fujita Health University Hospital Toyoake-shi Aichi-ken Recruiting
Tohoku University Hospital Sendai Miyagi Recruiting
National Center for Child Health and Development Tokyo Japan Active Not Recruiting
Erasmus MC Rotterdam South Holland Recruiting
Universitair Medisch Centrum Utrecht - PPDS Utrecht Netherlands Active Not Recruiting
King Faisal Specialist Hospital & Research Center - Riyadh Riyadh Ar Riya Active Not Recruiting
King Fahad Medical City Riyadh Ar Riya Not Yet Recruiting
King Abdullah Children's Specialist Hospital Riyadh Ar Riya Not Yet Recruiting
Hospital Sant Joan de Deu - PIN Esplugues de Llobregat Barcelona Active Not Recruiting
Hospital Universitario Cruces Barakaldo Biscay Recruiting
Hospital Universitario 12 de Octubre Madrid Spain Recruiting
Hospital Universitario Virgen del Rocio - PPDS Seville Spain Active Not Recruiting
University Hospital Birmingham NHS Foundation Trust Birmingham United Kingdom Recruiting
Birmingham Children's Hospital Birmingham United Kingdom Completed
Great Ormond Street Hospital for Children NHS Foundation Trust London United Kingdom Recruiting
Willink Biochemical Genetics Unit - PPDS Manchester United Kingdom Recruiting

More ModernaTX, Inc. trials in the UK

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04159103 on ClinicalTrials.gov ↗ ← All trials in the UK